Evaluation of Regional Treatment Effect in a Multiregional Clinical Trial

• Activity Number: 394
• Type: Topic Contributed
• Date/Time: Tuesday, August 3, 2010 : 2:00 PM to 3:50 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Yi Tsong* and W-J Chang and Chin-Fu Hsiao and Hsiao-Hui Tsou+
• Companies: CDER/FDA and National Health Research Institutes, Taiwan and National Health Research Institutes, Taiwan and National Health Research Institutes, Taiwan
• Keywords: Multi-regional ; region-specific ; consistency

Abstract

The primary objective of a multiregional bridging trial is to show the overall efficacy of a drug in all participating regions while also evaluating the possibility of applying the overall trial results to each region. In order to show that the results in that region be consistent with the overall results, we propose a few evaluation approaches to accommodate the sample size limitations. One of the approaches is to analyze a completed multi-regional trial for any specific regional effect by controlling the type I error rate adjusted for the regional sample size and the planned power of the global trial. On the other hand, in order to attain the approval for a specific region, the sample size requirement for any specific region is determined using the overall power planned and a maximum regional acceptable type I error rate.